A novel SMPD1 mutation in two Chinese sibling patients with type B Niemann-Pick disease

Chin Med J (Engl). 2012 Apr;125(8):1511-2.

Abstract

Type B Niemann-Pick disease is an autosomal recessive sphingolipidosis due to mutations in the sphingomyelin phosphodiesterase 1 gene (SMPD1). Here we present molecular findings for two sibling patients. One mutation V36A due to c.107T>C in exon 1 is a single nucleotide polymorphism and the other N522S due to c.1565 A>G in exon 6 is a novel missense mutation. This non-fatal missense mutation leads to –20% residual lysosomal acid sphingomyelinase activity in vitro and only results in hepatosplenomegaly without neurologic involvement.

Publication types

  • Case Reports

MeSH terms

  • Female
  • Humans
  • Middle Aged
  • Mutation, Missense*
  • Niemann-Pick Disease, Type B / genetics*
  • Polymorphism, Single Nucleotide*
  • Siblings
  • Sphingomyelin Phosphodiesterase / genetics*

Substances

  • SMPD1 protein, human
  • Sphingomyelin Phosphodiesterase